Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: Findings from the opinion-based PREDICT-FD modified Delphi consensus initiative
dc.contributor.author | Hughes, Derralynn A. | |
dc.contributor.author | Aguiar, Patricio | |
dc.contributor.author | Deegan, Patrick B | |
dc.contributor.author | Ezgu, Fatih | |
dc.contributor.author | Frustaci, Andrea | |
dc.contributor.author | Lidove, Olivier | |
dc.contributor.author | Linhart, Aleš | |
dc.contributor.author | Lubanda, Jean-Claude | |
dc.contributor.author | Moon, James C | |
dc.contributor.author | Nicholls, Kathleen | |
dc.contributor.author | Niu, Dau-Ming | |
dc.contributor.author | Nowak, Albina | |
dc.contributor.author | Ramaswami, Uma | |
dc.contributor.author | Reisin, Ricardo | |
dc.contributor.author | Rozenfeld, Paula | |
dc.contributor.author | Schiffmann, Raphael | |
dc.contributor.author | Svarstad, Einar | |
dc.contributor.author | Thomas, Mark | |
dc.contributor.author | Torra, Roser | |
dc.contributor.author | Vujkovac, Bojan | |
dc.contributor.author | Warnock, David G. | |
dc.contributor.author | West, Michael L | |
dc.contributor.author | Johnson, Jack | |
dc.contributor.author | Rolfe, Mark J | |
dc.contributor.author | Feriozzi, Sandro | |
dc.date.accessioned | 2021-04-29T11:24:34Z | |
dc.date.available | 2021-04-29T11:24:34Z | |
dc.date.created | 2020-11-11T13:03:59Z | |
dc.date.issued | 2020 | |
dc.Published | BMJ Open. 2020, 10:e035182 (10), 1-12. | |
dc.identifier.issn | 2044-6055 | |
dc.identifier.uri | https://hdl.handle.net/11250/2740367 | |
dc.description.abstract | Objectives The PRoposing Early Disease Indicators for Clinical Tracking in Fabry Disease (PREDICT-FD) initiative aimed to reach consensus among a panel of global experts on early indicators of disease progression that may justify FD-specific treatment initiation. Design and setting Anonymous feedback from panellists via online questionnaires was analysed using a modified Delphi consensus technique. Questionnaires and data were managed by an independent administrator directed by two non-voting cochairs. First, possible early indicators of renal, cardiac and central/peripheral nervous system (CNS/PNS) damage, and other disease and patient-reported indicators assessable in routine clinical practice were compiled by the cochairs and administrator from panellists’ free-text responses. Second, the panel scored indicators for importance (5-point scale: 1=not important; 5=extremely important); indicators scoring ≥3 among >75% of panellists were then rated for agreement (5-point scale: 1=strongly disagree; 5=strongly agree). Indicators awarded an agreement score ≥4 by >67% of panellists achieved consensus. Finally, any panel-proposed refinements to consensus indicator definitions were adopted if >75% of panellists agreed. Results A panel of 21 expert clinicians from 15 countries provided information from which 83 possible current indicators of damage (kidney, 15; cardiac, 15; CNS/PNS, 13; other, 16; patient reported, 24) were compiled. Of 45 indicators meeting the importance criteria, consensus was reached for 29 and consolidated as 27 indicators (kidney, 6; cardiac, 10; CNS/PNS, 2; other, 6; patient reported, 3) including: (kidney) elevated albumin:creatinine ratio, histological damage, microalbuminuria; (cardiac) markers of early systolic/diastolic dysfunction, elevated serum cardiac troponin; (CNS/PNS) neuropathic pain, gastrointestinal symptoms suggestive of gastrointestinal neuropathy; (other) pain in extremities/neuropathy, angiokeratoma; (patient-reported) febrile crises, progression of symptoms/signs. Panellists revised and approved proposed chronologies of when the consensus indicators manifest. The panel response rate was >95% at all stages. Conclusions PREDICT-FD captured global opinion regarding current clinical indicators that could prompt FD-specific treatment initiation earlier than is currently practised. | en_US |
dc.language.iso | eng | en_US |
dc.publisher | BMJ | en_US |
dc.rights | Navngivelse-Ikkekommersiell 4.0 Internasjonal | * |
dc.rights.uri | http://creativecommons.org/licenses/by-nc/4.0/deed.no | * |
dc.title | Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: Findings from the opinion-based PREDICT-FD modified Delphi consensus initiative | en_US |
dc.type | Journal article | en_US |
dc.type | Peer reviewed | en_US |
dc.description.version | publishedVersion | en_US |
dc.rights.holder | Copyright 2020 The Authors | en_US |
dc.source.articlenumber | e035182 | en_US |
cristin.ispublished | true | |
cristin.fulltext | original | |
cristin.qualitycode | 1 | |
dc.identifier.doi | 10.1136/bmjopen-2019-035182 | |
dc.identifier.cristin | 1846944 | |
dc.source.journal | BMJ Open | en_US |
dc.source.40 | 10:e035182 | |
dc.source.14 | 10 | |
dc.identifier.citation | BMJ Open. 2020, 10:e035182 | en_US |
dc.source.volume | 10 | en_US |
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